Survey of a treatment for congenital thrombotic thrombocytopenic purpura
Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)
This study is testing a new treatment called recombinant ADAMTS13 to see if it helps people with congenital thrombotic thrombocytopenic purpura feel better and stay healthy over 18 months.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 40 (estimated) |
| Sex | All |
| Sponsor | Takeda Industry-sponsored |
| Locations | 1 site (Tokyo) |
| Trial ID | NCT06441578 on ClinicalTrials.gov |
What this trial studies
This observational study surveys the use of recombinant ADAMTS13 in participants with congenital thrombotic thrombocytopenic purpura (cTTP) in Japan. Participants will receive intravenous injections of recombinant ADAMTS13 according to their clinic's standard practice, while the study will monitor for any side effects and assess the treatment's effectiveness in improving or preventing cTTP over an 18-month period. The study aims to gather data on the safety and efficacy of this treatment without direct involvement from the sponsor in patient care.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with congenital thrombotic thrombocytopenic purpura who are being treated with recombinant ADAMTS13.
Not a fit: Patients without congenital thrombotic thrombocytopenic purpura or those not receiving recombinant ADAMTS13 will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the safety and effectiveness of recombinant ADAMTS13 for patients with cTTP.
How similar studies have performed: While this approach is observational, similar studies on recombinant ADAMTS13 have shown promise in treating TTP, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: \- All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13 Exclusion Criteria: \- None
Where this trial is running
Tokyo
- Takeda selected site — Tokyo, Japan (Recruiting)
Study contacts
- Study coordinator: Takeda Contact
- Email: medinfoUS@takeda.com
- Phone: +1-877-825-3327
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.